Abstract
Background
The Pain, Functional Impairment, and Quality of Life study was an observational, cross-sectional assessment of the impact of pain on functional impairment and quality of life in adult people with hemophilia (PWH) of any severity in the USA who experience joint pain and/or bleeding.
Objective
To assess internal consistency (IC) and item-total correlation (ITC) of assessment tools used in the Pain, Functional Impairment, and Quality of Life study.
Methods
Participants completed 5 patient-reported outcome instruments (EQ-5D-5L with visual analog scale, Brief Pain Inventory v2 Short Form [BPI], International Physical Activity Questionnaire [IPAQ], Short Form 36 Health Survey v2 [SF-36v2], and Hemophilia Activities List [HAL]) and underwent an optional physiotherapist-administered musculoskeletal exam (Hemophilia Joint Health Score v2.1) during routine visits. Reliability assessment included IC and ITC of each instrument.
Results
A total of 381 adult PWH (median age, 34 years) were enrolled. Participants were predominantly white/non-Hispanic (69.2%); 75% had congenital hemophilia A, and 70.5% had severe hemophilia. A total of 310 subjects reported bleeding within the past 6 months (mean [SD] number of bleeds, 7.1 [13.00]). IC was generally high across the instruments employed (Cronbach’s alpha 0.79–0.98) with the exception of HAL use of transportation (0.58) and IPAQ total physical activity (0.51). ITC was high (Pearson’s product-moment correlation coefficient >0.20) for all items except the “vigorous intensity activities” item of IPAQ, which was applicable to less than one-third of participants. The ITCs were generally highest in domains/scores that measured the functional consequences of hemophilic arthropathy on mobility and pain.
Conclusion
The demonstrated reliability (IC/ITC) of the patient-reported outcome instruments and Hemophilia Joint Health Score v2.1 support a role for these instruments in evaluating adult PWH in US clinical and research settings.
Supplementary material
List of independent ethics committees/institutional review boards
Munson Medical Center, 1105 Sixth Street, Traverse City, MI 49684-2386, USA
Emory University, 1599 Clifton Road, 5th Floor, Atlanta, GA 30322, USA
St Vincent Hospital and Health Care Center, Inc., 8402 Harcourt Road, Suite 805, Indianapolis, IN 46260, USA
Western Institutional Review Board, 3535 7th Avenue SW, Olympia, WA 98502-5010, USA
Henry Ford Health System, CFP-Basement 046, 2799 West Grand Boulevard, Detroit, Ml 48202-2689, USA, Chairperson: Dr Timothy Roehrs, PhD
Vanderbilt University, 504 Oxford House, Nashville, TN 37232-4315, USA
Western Institutional Review Board, 1019 39th Avenue SE, Puyallup, WA 98374, USA
University of Colorado Multiple, Institutional Review Board, 13001 E. 17th Place, Building 500, Room N3214, Aurora, CO 80045, USA
Michigan State University, Olds Hall, 408 West Circle Drive, #207, East Lansing, MI 48824, USA, Chairperson: Dr Ashir Kumar
Oregon Health & Science University, 3181 SW Sam Jackson Park Road, Portland, OR 97239-3098, USA
University of Minnesota, D528 Mayo Memorial Building, 420 Delaware Street S.E., MMC 820, Minneapolis, MN 55455, USA
Rush University Medical Center, 1653 West Congress Parkway, Chicago, IL 60612-3833, USA
Wake Forest University Health Services, Medical Center Boulevard, Winston-Salem, NC 27157-1023, USA
Georgetown University, 37th and O Streets, N.W., Washington, DC 20057, USA
Acknowledgments
The P-FiQ study was managed by Quintiles Real World and Late Phase, Boston, Massachusetts, and Rockville, Maryland, with statistical analyses provided by Jennifer James, Senior Biostatistician, and was supported financially by Novo Nordisk Inc. Writing assistance was provided by Anna Abt, PhD, of ETHOS Health Communications, Yardley, Pennsylvania, and was supported financially by Novo Nordisk Inc., Plainsboro, New Jersey, in compliance with international Good Publication Practice guidelines. The abstract of this paper was presented at the 62nd Annual SSC Meeting of the ISTH as a poster presentation with interim findings. The poster’s abstract was published in the Journal of Thrombosis and Haemostasis: http://www.professionalabstracts.com/isth2016/programme-isth2016.pdf; DOI: 10.1111/jth.13325.
Disclosure
M Wang has served as a consultant to Baxalta, Biogen, CSL Behring, LFB, and Novo Nordisk Inc. K Batt has received grant/research support from Novo Nordisk Inc. and is a scientific advisor for Precision Health Economics. C Kessler has served on advisory boards for Baxalta, Bayer, Biogen, Genentech, Grifols, Novo Nordisk Inc., Octapharma, and Pfizer, and has received grant/research support from Baxter, Bayer, Novo Nordisk Inc., and Octapharma. A Neff has served on advisory boards for Baxalta, Kedrion, and Novo Nordisk Inc. NN Iyer is an employee of Novo Nordisk Inc. DL Cooper is an employee of Novo Nordisk Inc. CL Kemp-ton has served as a consultant to Baxalta, Genentech, and Novo Nordisk Inc. and has received grant/research support from Novo Nordisk Inc. The authors report no other conflicts of interest in this work.